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Journal Articles

Impact of Daratumumab-Containing Induction Therapy on Stem Cell Mobilization in Multiple Myeloma: A Retrospective, Single-Center Analysis

Hegta R. Figueiroa, Germano Medeiros, Juliana Matos Pessoa, Paula Lucafo Zenero, Isabella Pittol, Fauze Lutfe Ayoub, Pedro Paulo Faust Machado, Gabriela Camargo, Dimitra Oliveira Delijaicov, André Larubia, Arlett Edna Lazar, Jose Ulysses A. Filho, Breno Gusmao, Fabio R. Kerbauy, André Dias Americo, Phillip Scheinberg
Journal: Blood
Blood (2024) 144 (Supplement 1): 7262.
https://doi.org/10.1182/blood-2024-210191
Published: 2024
Journal Articles

Autologous Stem Cell Transplantation (ASCT) for Light Chain (AL) Amyloidosis: A Single-Center Report

Natalia Pin Chuen Zing, Breno Gusmao, Juliana Matos Pessoa, Jose Ulysses A. Filho, Fabio R. Kerbauy, Hegta R. Figueiroa, Isabella Pittol, Paula Lucafo Zenero, Phillip Scheinberg, Germano Lima, André Américo, Fauze Lutfe Ayoub
Journal: Blood
Blood (2024) 144 (Supplement 1): 6900.
https://doi.org/10.1182/blood-2024-211920
Published: 2024
Journal Articles

Oral Iptacopan Monotherapy Leads to Long-Term Improvements in Patient (Pt)-Reported Health-Related Quality of Life (HRQoL) and Investigator-Assessed Signs and Symptoms of Paroxysmal Nocturnal Hemoglobinuria (PNH): 48-Week (Wk) Results from the Phase III APPLY-PNH and APPOINT-PNH Trials

Antonio M Risitano, Bing Han, Austin G. Kulasekararaj, Yasutaka Ueda, Phillip Scheinberg, Carlos de Castro, Jaroslaw Maciejewski, Josefin Snellman, Olivier Somenzi, Randall Winnette, Samopriyo Maitra, Shujie Li, Marion Dahlke, Regis Peffault De Latour
Journal: Blood
Blood (2024) 144 (Supplement 1): 4079.
https://doi.org/10.1182/blood-2024-203532
Published: 2024
Journal Articles

Phase III COMMODORE 1 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Switched from Ravulizumab

Phillip Scheinberg, Hazza Alzahrani, Alejandro Contento Gonzalo, Martin Höglund, Jin Seok Kim, Jens Panse, Melissa J Rose, Jörg Schubert, Yasutaka Ueda, Eng Soo Yap, Muriel Buri, Nicolo' Compagno, Brittany Gentile, Sasha Sreckovic, Austin G. Kulasekararaj
Journal: Blood
Blood (2024) 144 (Supplement 1): 4078.
https://doi.org/10.1182/blood-2024-200889
Published: 2024
Journal Articles

Phase III COMMODORE 2 and 1 Trials: Characterization of Breakthrough Hemolysis Events in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab or Eculizumab

Austin G. Kulasekararaj, Jaroslaw Czyz, Catherine Flynn, Gabriela Roncone Gastal, Marek Hus, Chrysavgi Lalayanni, Erfan Nur, Phillip Scheinberg, Yasutaka Ueda, Alessandro Maria Vannucchi, Maria Vidriales Vicente, Iige Viigimaa, Simon Buatois, Alice C. Chang, Sammy Chebon, Alexander Röth
Journal: Blood
Blood (2024) 144 (Supplement 1): 1323.
https://doi.org/10.1182/blood-2024-200400
Published: 2024
Journal Articles

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease, Experience of a Brazilian Private Service

Hegta R. Figueiroa, Victor Gottardello Zecchin, Adriana Maria Paixao de Sousa da Silva, MArina Foltran Cela, Maria Lucia De Martino Lee, Fabricio Tera Romagnol, Fernanda Marinho Manga, Gabriela Camargo, Dimitra Oliveira Delijaicov, Leticia Santos Soares, Tomas Marzagão Barbuto, Ricardo Helman, Fabio R. Kerbauy, Phillip Scheinberg
Journal: Blood
Blood (2024) 144 (Supplement 1): 7322.
https://doi.org/10.1182/blood-2024-209967
Published: 2024
Journal Articles

The Effect of Oral Iptacopan Monotherapy on Hematological Parameters in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Is Consistent Regardless of the Type of Prior Anti-C5 Treatment Received: A Post Hoc Analysis of 24-Week Data from the Randomized Phase III APPLY-PNH Trial

Regis Peffault De Latour, Austin G. Kulasekararaj, Phillip Scheinberg, Yasutaka Ueda, Carlos de Castro, Samopriyo Maitra, Philippe Ferber, Marion Dahlke, Antonio M Risitano
Journal: Blood
Blood (2024) 144 (Supplement 1): 4087.
https://doi.org/10.1182/blood-2024-200763
Published: 2024
Journal Articles

Early Versus Standard Initiation of Immunosuppressive Therapy for Graft Versus Host Disease Prophylaxis in Haploidentical Allogeneic Stem Cell Transplantation: Incidence and Severity of Cytokine Release Syndrome and Related Outcomes

Luisa V. Carvalho, André Dias Americo, Camila Carminati Isoppo, Isabella Pittol, João A. G. G. Prats, Eurides L. Rosa, Germano Lima, Natalia Fumagalli, Paula Lucafo Zenero, Hegta R. Figueiroa, Fauze Lutfe Ayoub, Juliana Matos Pessoa, Jose Ulysses A. Filho, Fabio R. Kerbauy, Phillip Scheinberg
Journal: Blood
Blood (2024) 144 (Supplement 1): 7272.
https://doi.org/10.1182/blood-2024-193956
Published: 2024
Journal Articles

Phase III Randomized COMMODORE 1 Trial: 2-Year Safety and Efficacy of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Switched from Eculizumab

Phillip Scheinberg, Diego Villa Cle, Berangere Devalet, Valentina Giai, Cristina Barrenetxea Lekue, Hsuan-Yu Lin, Zsolt Nagy, Jun-ichi Nishimura, Jens Panse, Agnieszka Piekarska, Jörg Schubert, Kazutaka Sunami, Mustafa Yenerel, Anita Appius, Brittany Gentile, Shino Iwase, Himika Patel, Sasha Sreckovic, Austin G. Kulasekararaj
Journal: Blood
Blood (2024) 144 (Supplement 1): 1313.
https://doi.org/10.1182/blood-2024-200745
Published: 2024
Journal Articles

Incidence, Time to Diagnosis, and Therapeutic Landscape of Aplastic Anemia: Results from a Large United States Claims Database

Phillip Scheinberg, Tim H. H. Brummendorf, Regis Peffault De Latour, Carlo Dufour, Raj Gokani, Morag Griffin, Juliana Hey-Hadavi, Yoshinobu Kanda, Eric Mitchell, Barbara Możejko-Pastewka, Andres Quintero, Patryk Skowron, Meltem Kurt Yüksel, Alicia Rovó
Journal: Blood
Blood (2024) 144 (Supplement 1): 2329.
https://doi.org/10.1182/blood-2024-204908
Published: 2024
Journal Articles

Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III Apply-PNH and Appoint-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Regis Peffault De Latour, Austin Kulasekararaj, Phillip Scheinberg, Philippe Ferber, Tomasz Lawniczek, Christine Thorburn, Marion Dahlke, Antonio M Risitano
Journal: Blood
Blood (2023) 142 (Supplement 1): 1338.
https://doi.org/10.1182/blood-2023-179377
Published: 2023
Journal Articles

Data-Driven Harmonization of 2022 Who and ICC Classifications of Myelodysplastic Syndromes/Neoplasms (MDS): A Study By the International Consortium for MDS (icMDS)

Luca Lanino, Somedeb Ball, Jan Philipp Bewersdorf, Monia Marchetti, Giulia Maggioni, Erica Travaglino, Najla H Al Ali, Pierre Fenaux, Uwe Platzbecker, Valeria Santini, Maria Diez-Campelo, Avani M Singh, Akriti Gupta Jain, Luis E. Aguirre, Sara M. Tinsley-Vance, Zaker I. Schwabkey, Onyee Chan, Zhuoer Xie, Andrew M. Brunner, Andrew T. Kuykendall, John M. Bennett, Rena Buckstein, Rafael Bejar, Hetty E. Carraway, Amy E. DeZern, Elizabeth A. Griffiths, Stephanie Halene, Robert Hasserjian, Jeffrey Lancet, Alan F. List, Sanam Loghavi, Olatoyosi Odenike, Eric Padron, Mrinal M. Patnaik, Gail J. Roboz, Maximilian Stahl, Mikkael A. Sekeres, David Peter Steensma, Michael R. Savona, Justin Taylor, Mina Xu, Kendra Sweet, David A Sallman, Stephen D. Nimer, Christopher S. Hourigan, Andrew H. Wei, Elisabetta Sauta, Saverio D'Amico, Gianluca Asti, Gastone Castellani, Uma M. Borate, Guillermo Sanz, Fabio Efficace, Steven D. Gore, Tae Kon Kim, Naval Daver, Guillermo Garcia-Manero, María Rozman, Alberto Orfao, Sa A. Wang, M. Kathy Foucar, Ulrich Germing, Torsten Haferlach, Phillip Scheinberg, Yasushi Miyazaki, Marcelo Iastrebner, Austin Kulasekararaj, Thomas Cluzeau, Shahram Kordasti, Arjan A. van de Loosdrecht, Lionel Ades, Amer M. Zeidan, Rami S. Komrokji, Matteo Giovanni Della Porta
Journal: Blood
Blood (2023) 142 (Supplement 1): 998.
https://doi.org/10.1182/blood-2023-186580
Published: 2023
Journal Articles

Patient-Reported Improvements in Fatigue and Health-Related Quality of Life in the Phase 3 Studies Apply-PNH and Appoint-PNH Evaluating the Use of Iptacopan in C5 Inhibitor-Treated and Treatment-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria

Antonio M Risitano, Carlos M de Castro, Bing Han, Austin Kulasekararaj, Jaroslaw P. Maciejewski, Phillip Scheinberg, Yasutaka Ueda, Susan Vallow, Georgina Bermann, Marion Dahlke, Rakesh Kumar, Regis Peffault de Latour
Journal: Blood
Blood (2023) 142 (Supplement 1): 487.
https://doi.org/10.1182/blood-2023-179035
Published: 2023
Journal Articles

Safety of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Pooled Results from the Phase III COMMODORE 1, COMMODORE 2, and COMMODORE 3 Studies

Alexander Roeth, Rong FU, Guangsheng He, Hazza A Alzahrani, Sheng-Chieh Chou, Yosr Hicheri, Maciej Kazmierczak, Viviane Lacorte Recova, Michihiro Uchiyama, ANA Maria Vladareanu, Leigh Beveridge, Simon Buatois, Muriel Buri, Dayu Shi, Nadiesh Balachandran, Sasha Srekovic, Phillip Scheinberg
Journal: Blood
Blood (2023) 142 (Supplement 1): 575.
https://doi.org/10.1182/blood-2023-177618
Published: 2023
Journal Articles

Factor B Inhibition with Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from the Multicenter, Phase III APPLY-PNH Trial

Antonio M Risitano, Austin Kulasekararaj, Alexander Roeth, Phillip Scheinberg, Yasutaka Ueda, Carlos de Castro, Eros Di Bona, Morag Griffin, Saskia MC Langemeijer, Hubert Schrezenmeier, Wilma Barcellini, Vitor AQ Mauad, Jens Panse, Philippe Schafhausen, Suzanne Tavitian, Eloise Beggiato, Anna Gaya, Wei-Han Huang, Toshio Kitawaki, Abdullah Kutlar, Jaroslaw P. Maciejewski, Rosario Notaro, Vinod Pullarkat, Jörg Schubert, Louis Terriou, Michihiro Uchiyama, Flore Sicre De Fontbrune, Camilla Frieri, Ferras Alashkar, Shreyans Gandhi, Rakesh Kumar, Christine Thorburn, Samopriyo Maitra, Susan Solar-Yohay, Tomasz Lawniczek, Marion Dahlke, Régis Peffault De Latour
Journal: Blood
Blood (2023) 142 (Supplement 1): 571.
https://doi.org/10.1182/blood-2023-180780
Published: 2023
Journal Articles

Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Naïve Patients in the Phase III APPOINT-PNH Trial

Antonio M Risitano, Bing Han, Austin Kulasekararaj, Yasutaka Ueda, Phillip Scheinberg, Carlos de Castro, Jaroslaw P. Maciejewski, Josefin Snellman, Ranjan Tiwari, Marion Dahlke, Régis Peffault de Latour
Journal: Blood
Blood (2023) 142 (Supplement 1): 4084.
https://doi.org/10.1182/blood-2023-180866
Published: 2023
Journal Articles

Patient Experience Interviews with a Novel Treatment Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

Carlos De Castro, Phillip Scheinberg, Bing Han, Susan Vallow, Georgina Bermann, Marion Dahlke, Zhixin Wang, Rakesh Kumar, Gavin Dickie, Caitlyn Lowe, Kaelyn Rupinski, Amber Nieves, Regis Peffault De Latour
Journal: Blood
Blood (2023) 142 (Supplement 1): 7339.
https://doi.org/10.1182/blood-2023-186840
Published: 2023
Journal Articles

Patient Preferences and Treatment Satisfaction in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Approved C5 Inhibitors in the Phase III Randomized COMMODORE 1 and COMMODORE 2 Trials

Austin Kulasekararaj, Fernando Ataulfo Gonzalez Fernandez, Phillip Scheinberg, Nicole Straetmans, Yasutaka Ueda, Brittany Gentile, Jennifer Stefani, Marianne Uguen, Alexander Roeth
Journal: Blood
Blood (2023) 142 (Supplement 1): 5628.
https://doi.org/10.1182/blood-2023-178610
Published: 2023
Journal Articles

Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes

Clinical Trials & Observations
Amer M. Zeidan, Uwe Platzbecker, Jan Philipp Bewersdorf, Maximilian Stahl, Lionel Adès, Uma Borate, David Bowen, Rena Buckstein, Andrew Brunner, Hetty E. Carraway, Naval Daver, Maria Díez-Campelo, Theo de Witte, Amy E. DeZern, Fabio Efficace, Guillermo Garcia-Manero, Jacqueline S. Garcia, Ulrich Germing, Aristoteles Giagounidis, Elizabeth A. Griffiths, Robert P. Hasserjian, Eva Hellström-Lindberg, Marcelo Iastrebner, Rami Komrokji, Austin G. Kulasekararaj, Luca Malcovati, Yasushi Miyazaki, Olatoyosi Odenike, Valeria Santini, Guillermo Sanz, Phillip Scheinberg, Reinhard Stauder, Arjan A. van de Loosdrecht, Andrew H. Wei, Mikkael A. Sekeres, Pierre Fenaux
Journal: Blood
Blood (2023) 141 (17): 2047–2061.
https://doi.org/10.1182/blood.2022018604
Published: 2023
Includes: Supplemental data
Journal Articles

Oral Monotherapy with Iptacopan, a Proximal Complement Inhibitor of Factor B, Has Superior Efficacy to Intravenous Terminal Complement Inhibition with Standard of Care Eculizumab or Ravulizumab and Favorable Safety in Patients with Paroxysmal Nocturnal Hemoglobinuria and Residual Anemia: Results from the Randomized, Active-Comparator-Controlled, Open-Label, Multicenter, Phase III Apply-PNH Study

Regis Peffault de Latour, Alexander Roeth, Austin Kulasekararaj, Phillip Scheinberg, Yasutaka Ueda, Carlos M de Castro, Eros Di Bona, Hubert Schrezenmeier, Saskia MC Langemeijer, Wilma Barcellini, Suzanne Tavitian, Jens Panse, Philippe Schafhausen, Vitor AQ Mauad, Cecile Kerloeguen, Rafael Levitch, Rakesh Kumar, Christine Thorburn, Samopriyo Maitra, Marion Dahlke, Antonio M Risitano
Journal: Blood
Blood (2022) 140 (Supplement 2): LBA-2.
https://doi.org/10.1182/blood-2022-171469
Published: 2022